Additional Relevant Details The phase I/II clinical trial is an open-label, dose-ranging study designed to treat up to 18 naïve male and female adult patients.  The three dose cohorts include 0.2 mg/kg, 1mg/kg and 2mg/kg with intravenous infusions of PRX-102 every two weeks.
The efficacy and safety analysis of the phase I/II clinical trial includes data from all 16 enrolled patients (9 male and 7 female) who completed the 12-month treatment period and, of more importance, a subset analysis of 10 patients who met the classic Fabry disease criteria (9 male and 1 female). The classic Fabry patient population is the most common Fabry patient population studied and reported on in the scientific and medical literature, and is the population to be evaluated in the Company’s phase III pivotal trial.  Classic Fabry disease is characterized by having less than 30% residual enzyme activity, and typical manifestations involve neurological, skin, ophthalmic and Fabry-specific biomarkers.